Sobi and Biogen success in Phase III

27.09.2012 - Swedish Sobi and Biogen Idec have proved safety and efficacy of a haemophila B drug that prevents bleeding three times longer than other clotting factors.

In the B-LONG Phase III study on 126 haemophila B patients, the partners proved safety and efficacy of their long-lasting recombinant Factor IX Fc fusion protein (rFIXFc). According to the companies, 90.4% of bleeding episodes were controlled by a single shot of the engineered clotting factor and showed only one serious adverse effect possibly related to the product. Biogen Idec plans to submit a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) in the first half of 2013. Filing in Europe will require completion of the ongoing Kids B-LONG study in children less than 12 years.

The top line results are good news for the Swedish drug maker, which had to restructure last year due to loss in revenues. According to pharmacokinetic (PK) analysis terminal half-life of the fusion protein of Factor IX and the Fc domain of human IgG(1) was 82 hours compared to 34 hours for BeneFIX.“Our companies are pioneering the application of Fc fusion technology to extend the half-life of clotting factors. Fc fusion technology utilises a naturally-occurring recycling pathway that has been successfully employed in other therapeutic areas. This approach holds promise for combining more consistent protection with fewer injections,” said Sobi CEO Geoffrey McDonough.

The B-LONG study had four treatment arms: weekly prophylaxis, individualised interval prophylaxis, episodic treatment and perioperative management (Arms 1, 2, 3 and 4, respectively). 93.5 percent of patients completed the study. Recombinant FIXFc was generally well-tolerated. No inhibitors to rFIXFc were detected and no cases of anaphylaxis were reported in any patients. The overall median annualised bleeding rates (including spontaneous and traumatic bleeds) were 2.95 in the weekly prophylaxis arm, 1.38 in the individualised interval prophylaxis arm, and 17.69 in the episodic treatment arm. In the individualised interval prophylaxis arm, the median dosing interval during the last 6 months on study was 14 days.

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